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First-Line Therapy Options for Patients with ITP

Panelists: Ivy Altomare, MD, Duke University Medical Center; Ralph V. Boccia, MD, FACP, LLC, Georgetown University Medical Center; Amit Mehta, MD Independent Hematology and Oncology Practice
Published: Tuesday, Jan 29, 2019



Transcript: 

Ivy Altomare, MD: Absolutely. OK, so let’s move on to treatment, first-line treatment. How do you approach first-line treatment?

Amit Mehta, MD: Well, I think it’s a good segue, because for first-line treatment for acute ITP, it’s a little bit more straightforward as far as the general category of using corticosteroid agents, whereas in the relapsed ITP or persistent or chronic ITP setting, of course, it’s much more complex as far as your treatment decision, algorithm, and adverse effect discussion and response rates, etc. Really, the main approach is that, OK, well, the patient has a platelet count low enough that warrants treatment or they’re having symptomatology or both that warrants treatment and, therefore, we need to treat the patient and stabilize them. So, conversely, is that there are some patients who had ITP, it should be said, who may have a milder platelet count decrease that may not necessarily warrant treatment. There are some people who have a platelet count, as we know—maybe 75,000—and they may be ITP-immune thrombocytopenia in our workup, but they may not have any indications that they need therapy.

Ivy Altomare, MD: Absolutely. If you look at registry data, it’s 50% of patients who can be observed. But if you do need to start, what do you use first line?

Amit Mehta, MD: Yeah, so the 2 biggest choices generally will be that a clinician will consider a long-term prednisone taper where 1 mg/kg of prednisone is started orally daily and then gradually tapered over several weeks, which is more of a traditional type of mode of therapy. And then what’s been used more in recent years more commonly is pulsed-dose dexamethasone—so, dexamethasone 4 straight days, 40 mg a day, and then potentially repeating that 7 to 14 days later if you’re not satisfied with the response.

So, head-to-head studies between the pulsed-dose, higher-dose dexamethasone versus the traditional prednisone route have shown higher response rates with the dexamethasone arm, as well as better durability of response over time—so, that’s 1 reason that that treatment protest came to the fore a little bit more in recent years—and the other added benefit of cutting out the long taper of prednisone, which was often a real issue for patients in terms of possible additive adverse effects, whether you talk about things like headaches and sugar changes and hypertension and sleep disturbance and appetite changes, etc. So it’s nice to give a shorter course of steroids with a higher response rate, it seems from clinical trials, and that also results in good durability of response for these patients. So usually…my approach is that I’ll typically use the 4-day dexamethasone arm, preferentially for the majority of patients…I treat for acute ITP.

Ivy Altomare, MD: I’m the same. I have switched from prednisone to high-dose dexamethasone. I do find that it works just as well and it is a little bit better tolerated. But, of course, everybody is a little bit different. How about you, what do you do?

Ralph V. Boccia, MD, FACP, LLC: I do the same—dex [dexamethasone]. I’ve been doing dex for probably the last 2 or 3 years.

Ivy Altomare, MD: What about if a patient is critically ill or bleeding, very heavily bleeding?

Ralph V. Boccia, MD, FACP, LLC: I think all of us today would pull out all stops, and so we would try to block destruction with IVIG [intravenous immunoglobulin], high-dose immunoglobulin. Start the patient on a course of corticosteroids, and if they’re bleeding, also give them platelets. It doesn’t help much to give platelets unless you’re giving IVIG and you can block the Fc receptor on the macrophage, but if you do all 3 of them, most patients will respond to that. So in the critically ill patient, I think the triplet therapy is the standard of care.

Amit Mehta, MD: Yeah, I would definitely agree…that you want to pull out all the stops for the acutely bleeding patient whether it’s a hospital situation, ICU [intensive care unit] situation, perioperative type of situation. And for the clinicians in practice, it should be known that the platelet life span for the transfused platelets will be very short, so that is the critical point—that giving IVIG in conjunction is useful to try to maximize the benefit, knowing that the kinetics of the platelet response will be short-lived anyway, but at least in that short-lived duration, pulling out all stops might be enough to stop the bleeding and achieving hemostasis, at least to some degree.

Ivy Altomare, MD: Sure.

Ralph V. Boccia, MD, FACP, LLC: So 2 further points: One is to make sure you give the IVIG first.

Amit Mehta, MD: Correct.

Ralph V. Boccia, MD, FACP, LLC: And the second is just to think about the mechanism of action, the response rate, and the response timing that will be within, we know, 24 to 48 hours after giving IVIG. If they’re going to respond, they will respond to it, where steroids take almost a week. So you have almost built into that sequential therapies that will carry and build on each other and be somewhat synergistic.

Ivy Altomare, MD: Right. But, although IVIG induces a 75% to 90% response rate, the response rate is lost quickly—typically, in the second week. So it’s good to have the steroids on board, as well.

Transcript Edited for Clarity 

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Transcript: 

Ivy Altomare, MD: Absolutely. OK, so let’s move on to treatment, first-line treatment. How do you approach first-line treatment?

Amit Mehta, MD: Well, I think it’s a good segue, because for first-line treatment for acute ITP, it’s a little bit more straightforward as far as the general category of using corticosteroid agents, whereas in the relapsed ITP or persistent or chronic ITP setting, of course, it’s much more complex as far as your treatment decision, algorithm, and adverse effect discussion and response rates, etc. Really, the main approach is that, OK, well, the patient has a platelet count low enough that warrants treatment or they’re having symptomatology or both that warrants treatment and, therefore, we need to treat the patient and stabilize them. So, conversely, is that there are some patients who had ITP, it should be said, who may have a milder platelet count decrease that may not necessarily warrant treatment. There are some people who have a platelet count, as we know—maybe 75,000—and they may be ITP-immune thrombocytopenia in our workup, but they may not have any indications that they need therapy.

Ivy Altomare, MD: Absolutely. If you look at registry data, it’s 50% of patients who can be observed. But if you do need to start, what do you use first line?

Amit Mehta, MD: Yeah, so the 2 biggest choices generally will be that a clinician will consider a long-term prednisone taper where 1 mg/kg of prednisone is started orally daily and then gradually tapered over several weeks, which is more of a traditional type of mode of therapy. And then what’s been used more in recent years more commonly is pulsed-dose dexamethasone—so, dexamethasone 4 straight days, 40 mg a day, and then potentially repeating that 7 to 14 days later if you’re not satisfied with the response.

So, head-to-head studies between the pulsed-dose, higher-dose dexamethasone versus the traditional prednisone route have shown higher response rates with the dexamethasone arm, as well as better durability of response over time—so, that’s 1 reason that that treatment protest came to the fore a little bit more in recent years—and the other added benefit of cutting out the long taper of prednisone, which was often a real issue for patients in terms of possible additive adverse effects, whether you talk about things like headaches and sugar changes and hypertension and sleep disturbance and appetite changes, etc. So it’s nice to give a shorter course of steroids with a higher response rate, it seems from clinical trials, and that also results in good durability of response for these patients. So usually…my approach is that I’ll typically use the 4-day dexamethasone arm, preferentially for the majority of patients…I treat for acute ITP.

Ivy Altomare, MD: I’m the same. I have switched from prednisone to high-dose dexamethasone. I do find that it works just as well and it is a little bit better tolerated. But, of course, everybody is a little bit different. How about you, what do you do?

Ralph V. Boccia, MD, FACP, LLC: I do the same—dex [dexamethasone]. I’ve been doing dex for probably the last 2 or 3 years.

Ivy Altomare, MD: What about if a patient is critically ill or bleeding, very heavily bleeding?

Ralph V. Boccia, MD, FACP, LLC: I think all of us today would pull out all stops, and so we would try to block destruction with IVIG [intravenous immunoglobulin], high-dose immunoglobulin. Start the patient on a course of corticosteroids, and if they’re bleeding, also give them platelets. It doesn’t help much to give platelets unless you’re giving IVIG and you can block the Fc receptor on the macrophage, but if you do all 3 of them, most patients will respond to that. So in the critically ill patient, I think the triplet therapy is the standard of care.

Amit Mehta, MD: Yeah, I would definitely agree…that you want to pull out all the stops for the acutely bleeding patient whether it’s a hospital situation, ICU [intensive care unit] situation, perioperative type of situation. And for the clinicians in practice, it should be known that the platelet life span for the transfused platelets will be very short, so that is the critical point—that giving IVIG in conjunction is useful to try to maximize the benefit, knowing that the kinetics of the platelet response will be short-lived anyway, but at least in that short-lived duration, pulling out all stops might be enough to stop the bleeding and achieving hemostasis, at least to some degree.

Ivy Altomare, MD: Sure.

Ralph V. Boccia, MD, FACP, LLC: So 2 further points: One is to make sure you give the IVIG first.

Amit Mehta, MD: Correct.

Ralph V. Boccia, MD, FACP, LLC: And the second is just to think about the mechanism of action, the response rate, and the response timing that will be within, we know, 24 to 48 hours after giving IVIG. If they’re going to respond, they will respond to it, where steroids take almost a week. So you have almost built into that sequential therapies that will carry and build on each other and be somewhat synergistic.

Ivy Altomare, MD: Right. But, although IVIG induces a 75% to 90% response rate, the response rate is lost quickly—typically, in the second week. So it’s good to have the steroids on board, as well.

Transcript Edited for Clarity 
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